Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2022;9(4):463-476.
doi: 10.3233/JND-210776.

Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy

W Douglas Biggar  1 , Andrew Skalsky  2 , Craig M McDonald  3
Affiliations
Review

Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy

W Douglas Biggar et al. J Neuromuscul Dis. 2022.

Abstract

Deflazacort and prednisone/prednisolone are the current standard of care for patients with Duchenne muscular dystrophy (DMD) based on evidence that they improve muscle strength, improve timed motor function, delay loss of ambulation, improve pulmonary function, reduce the need for scoliosis surgery, delay onset of cardiomyopathy, and increase survival. Both have been used off-label for many years (choice dependent on patient preference, cost, and geographic location) before FDA approval of deflazacort for DMD in 2017. In this review, we compare deflazacort and prednisone/prednisolone in terms of their key pharmacological features, relative efficacy, and safety profiles in patients with DMD. Differentiating features include lipid solubility, pharmacokinetics, changes in gene expression profiles, affinity for the mineralocorticoid receptor, and impact on glucose metabolism. Evidence from randomized clinical trials, prospective studies, meta-analyses, and post-hoc analyses suggests that patients receiving deflazacort experience similar or slower rates of functional decline compared with those receiving prednisone/prednisolone. Regarding side effects, weight gain and behavior side effects appear to be greater with prednisone/prednisolone than with deflazacort, whereas bone health, growth parameters, and cataracts appear worse with deflazacort.

Keywords: Deflazacort; duchenne muscular dystrophy; glucocorticoids; prednisolone; prednisone.

PubMed Disclaimer

Conflict of interest statement

W. Douglas Biggar, MD has served as a consultant for PTC Therapeutics and Marathon.

Andrew Skalsky, MD has no conflicts of interest to report.

Craig M. McDonald, MD has served as a consultant for clinical trials for PTC Therapeutics, and outside the submitted work with Astellas Pharma, Avidity Biosciences, Capricor Therapeutics, Catabasis Pharmaceuticals, Edgewise Therapeutics, Entrada Therapeutics, Epirium Bio (formerly Cardero Therapeutics), FibroGen, Italfarmaco, Pfizer, PTC Therapeutics, Roche, Santhera Pharmaceuticals and Sarepta Therapeutics. He has received research support for clinical trials from Capricor Therapeutics, Catabasis Pharmaceuticals, Italfarmaco, Pfizer, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics. He serves on external advisory boards related to Duchenne muscular dystrophy for PTC Therapeutics, Edgewise Therapeutics, Eli Lilly, Sarepta Therapeutics, Santhera Pharmaceuticals, and Capricor Therapeutics.

The authors received no compensation for writing this article.

Figures

Fig. 1
Fig. 1
Structures of prednisone, prednisolone, and deflazacort, as listed in the PubChem database [70].
See this image and copyright information in PMC

References

    1. Mendell JR, Shilling C, Leslie ND, Flanigan KM, al-Dahhak R, Gastier-Foster J, et al..Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71(3):304–13. - PubMed
    1. Moat SJ, Bradley DM, Salmon R, Clarke A, Hartley L. Newborn bloodspot screening for Duchenne muscular dystrophy: 21 years experience in Wales (UK). Eur J Hum Genet. 2013;21(10):1049–53. - PMC - PubMed
    1. Ryder S, Leadley RM, Armstrong N, Westwood M, de Kock S, Butt T, et al.. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: An evidence review. Orphanet J Rare Dis. 2017;12(1):79. - PMC - PubMed
    1. Monaco AP, Neve RL, Colletti-Feener C, Bertelson CJ, Kurnit DM, Kunkel LM. Isolation of candidate cDNAs for portions of the Duchenne muscular dystrophy gene. Nature323. 1986;323(6089):646–50. - PubMed
    1. Pasternak C, Wong S, Elson EL. Mechanical function of dystrophin in muscle cells. J Cell Biol. 1995;128(3):355–61. - PMC - PubMed